The Orphan Products Development (OPD) program was created to identify and promote the development of orphan products (drugs, biologics, medical devices, and medical foods) that are indicated for a rare disease or condition. The term “rare disease or condition” is defined in 21 U.S.C. 360ee. As a practical way to implement the statutory definition, for devices and foods as well as for drugs, FDA considers drugs, devices, and medical foods potentially eligible for grants under the OPD grant program if they are indicated for a disease or condition that has a prevalence, not incidence, of fewer than 200,000 people in the United States.
The goal of this grant program is to support the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA provides grants for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products. Applicants must include in the application’s Background and Significance section documentation to support that the estimated prevalence of the orphan disease or condition in the United States is less than 200,000 (or in the case of a vaccine or diagnostic, information to support that the product will be administered to fewer than 200,000 people in the United States per year),and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development.
Additional information may be required upon request, for example, regarding population estimate and rationale. This additional information may be required, in part, to assure that human clinical trials of drugs are eligible to receive funding under the OPD grant program. 21 U.S.C. 360ee(b)(1)(A).See Section VIII, Other Information – Required Federal Citations, for policies related to this announcement.
New applications: February 4, 2015; February 3, 2016; February 1, 2017; February 7, 2018 by 11:59 PM Eastern Time
Resubmissions: October 15, 2015; October 14, 2016; October 16, 2017; October 15, 2018) by 11:59 PM Eastern Time
Filed Under: Funding Opportunities